Tuesday, 31 March 2020

EU GMP Annex 1: What The ‘Final’ Draft Reveals


A new paper reviews the new draft of EU GMP Annex 1. In doing so the focus is on those aspects that are different to the 2017 draft, rather than spending much time comparing the 2020 draft with the current Annex 1 (which is dated 2009).

The review includes an overview of the core changes, which include:

The global acceptance and implementation of ICH Q9 (Quality Risk Management)  and Q10 (Pharmaceutical Quality System), is not reflected in the current Annex. The new draft contains many references to Quality Risk Management (QRM) in particular, emphasizing that QRM should be used as a proactive tool. There are now 92 instances of the word “risk” in the new draft, an increase from 20 in the previous version.

There have been advances in sterile manufacturing technology, especially with RABS and isolators. There have also been advances with rapid microbiological methods, which the draft Annex acknowledges.

There was some ambiguity with the current version and these needed correction or clarification.

Annex 1 is often beyond sterile manufacturing, including aspects of non-sterile manufacturing. The scope of the new draft has been modified to reflect this.

There is the requirement for a formal, holistic contamination control strategy (which is now abbreviated to ‘CCS’ in the new draft). The expectation now appears to be for a formal document which reflects the site-wide strategy for minimizing contamination control with respect to sterile manufacturing .


The requirements of the contamination control strategy have been widened (43 mentions, up from 19 in the 2017 draft), however, with the new draft, extending to the need to fully-understand and review design, procedural, technical and organizational controls.

The reference is:

Sandle, T. (2020) EU GMP Annex 1: What The ‘Final’ Draft Reveals, Journal of GXP Compliance, 24 (2): 1-15, at: https://www.ivtnetwork.com/article/eu-gmp-annex-1-what-%E2%80%98final%E2%80%99-draft-reveals

For details, contact Tim Sandle

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

Monday, 30 March 2020

Guide to sterility test isolators


An isolator is an arrangement of physical barriers that are integrated so that the workspace (an enclosed environment) within the isolator is sealed from the outside environment. Isolators provide a testing environment free from contamination, through routine sanitization using a validated cycle and confirmed by environmental monitoring.

In addition, these devices enable the isolation between the operator and the process. There are many complications with isolators, from design to qualification, and with general operation.

It certainly remains that isolators cannot prevent contamination caused by GMP deficiencies such as poor aseptic procedures and inadequate training of operators.

To address these concerns and to outline best practices, Tim Sandle has written a new Pharmig guide:

Sandle, T. (2019) Guide to sterility test isolators, Pharmig, Stanstead Abbotts: UK (ISBN 978-0-9560804-9-3)

Foe details see: https://www.pharmig.org.uk/en/products/publications/

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

Sunday, 29 March 2020

Qualification of GC Equipment


EDQM has posted new guidance on the qualification of gas chromatography equipment.

The document is the second Annex of the core document “Qualification of Equipment”, and it should be used in combination with it when planning, performing and documenting the GC equipment qualification process.

The core document contains the general introduction and the Level I and II of qualification, common to all type of instruments, and the present annex contains GC instrument-related recommendations on parameters to be checked and the corresponding typical acceptance limits, as well as practical examples on the methodology that can be used to carry out these checks.

Level III (Periodic and motivated instrument calibration/checks) and IV (In-use instrument checks) qualifications must be carried out as an ISO 17025 requirement.

The tests proposed in the Level III and IV of qualification are based on an overall approach, in which several parameters are checked at the same time in a combined test procedure, to obtain information on the overall system performance (e.g. peak area precision, retention time precision, temperature programme reproducibility, etc). Nevertheless, it should be noted that it is also acceptable to check these parameters individually by using other well-defined procedures.

Requirements and (if applicable) corresponding typical acceptance limits (given in bold) should be applied; however other appropriately justified approaches are acceptable. Exemplary procedures provided in this document have non-binding character. They can be helpful when carrying out the required qualification.

Nevertheless, it is left to the professional judgement and background experience of each OMCL to decide on the most relevant procedures to be undertaken in order to provide evidence that their GC systems are working properly and are suitable for their intended use. If the qualification of equipment is done by the manufacturer or an external service provider, it is the responsibility of the OMCL to make sure that this is in line with the requirements set out in this guideline.

For details, see EDQM

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

Saturday, 28 March 2020

Update for testing for particulate contamination


At its 165th session in November 2019, the European Pharmacopoeia Commission adopted two general chapters related to testing for particulate contamination in pharmaceutical preparations.

The revisions to general chapter 2.9.19. Particulate contamination: sub-visible particles supplement the Pharmacopoeial Discussion Group (PDG) harmonised text with alternative local requirements applicable to biological parenteral preparations. Such preparations are provided in low volumes and the local requirements – marked in the text with white diamonds – allow testing of these and other preparations to be performed using volumes smaller than 5 mL where suitable instrumentation is available. The PDG remains committed to further revising the chapter in order to integrate these changes into the harmonised text.

The new, non-mandatory general chapter 5.17.2. Recommendations on testing of particulate contamination: visible particles provides information on visual inspection and control of visible particles in liquid preparations for which testing according to the general chapter 2.9.20. Particulate contamination: visible particles applies. The text highlights the different sources of foreign particle contamination of liquid preparations and the fact that every effort should be made to avoid their presence. Consideration is given to the different inspection stages during production and quality control, including stability testing. Acceptable quality level (AQL) testing (with reference to ISO standard 2859-1) following a 100% inspection of the batch is also addressed. This chapter is not intended to elaborate on GMP requirements, but rather should be read in conjunction with them. Detection of visible particles in parenteral products is probabilistic in nature and the occurrence of particles is random. The chapter therefore provides guidance on how users can establish that their product is “practically free from particles”.

These two chapters will be published in European Pharmacopoeia (Ph. Eur.) Supplement 10.3 and will become effective on 1 January 2021.


For further details see: Ph. Eur.

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

Friday, 27 March 2020

Gut Microbiome Samples Head for Space


Los Alamos National Laboratory scientists Armand Dichosa and Anand Kumar have sent samples of the human gut microbiome into space tonight, part of a project with NASA, DTRA and Rhodium Scientific.

On its 11:50 p.m. March 6 launch, SpaceX-20 they carried these samples to the International Space Station National Laboratory where they will be allowed to grow in order to understand the effect microgravity has on the microbial community. The samples have been carefully prepared at Los Alamos so that for each sample there is a complementary sample staying here on Earth.

Genomics Investigation of Human Gut Microbiome to Determine Effects of Microgravity Exposures (Rhodium Space Microbiome) examines the effects of spaceflight on the human gut microbiome, a complex community of numerous bacterial species. Developing a better understanding shifts in microbiome diversity and function and how they affect human health and performance may help protect people on future missions. Recent studies have shown a connection between alterations in the structure and function of the gut microbiome and multiple chronic and acute diseases.
Upon return, both sets of samples will be sequenced and analyzed to identify genetic changes that might occur to the bacterial communities, and to understand the potential implications these changes may have to human health in microgravity.

NASA has a video of the live-stream the March 6 launch at 11:50 p.m. Eastern Time

See: More information on the experiment

Also check-out:

Social Media handles - 

#LosAlamosNatLab
#BusinessOfScience
#SpaceMicrobiome

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

Thursday, 26 March 2020

CBD: It's (Biologically) More Intricate Than You Think

Image Source: Unsplash
In many ways, the hemp plant is intertwined with the history of the United States. British settlers even brought a store of hemp seeds along during their journey across the Atlantic and were “compelled by law to grow hemp.” Today, we understand that hemp has numerous medical benefits, but during colonial times, hemp was primarily used in textile and fiber-based applications, at least until 1937.

A guest post by Indiana Lee
That year, Congress passed the Marihuana Tax Act, which effectively made hemp cultivation a crime. At the time, hemp was considered dangerous, lumped in with its cousin marijuana, which has psychoactive effects when ingested or smoked. Yet the hemp plant contains only trace amounts of tetrahydrocannabinol (THC), the cannabinoid that’s responsible for a marijuana user’s “high.”

While, as the most notorious of the estimated 113 cannabinoids found in the cannabis plant, THC gets the bulk of press surrounding the plant, it’s not necessarily the star of the show. For instance, cannabidiol, commonly known as CBD, doesn’t induce euphoria but has shown promise in treating a wide variety of health conditions, from anxiety and insomnia to Alzheimer’s and neurodegenerative disorders.

For this reason, and thanks to the 2018 passage of the Hemp Farming Act of 2018 (H.R.5485), which re-legalized hemp cultivation at the federal level, CBD shops are popping up in strip malls and co-ops across the country. We still have more to learn about CBD, however, and consumers should note that not all products containing CBD are consistently effective.

Like its cousin marijuana, the cellular makeup of hemp can vary considerably between strains, resulting in differing effects depending on an individual's condition and the plant’s potency. In effect, the idea of “CBD” has been oversimplified for the masses, but it’s much more complex than meets the eye.

Cannabis Legalization and Terminology

Fast forward to the 21st century, where, after decades of prohibition, the hemp industry is once again booming. The passage of H.R. 5485 paved the way towards a greater understanding of and research on the ways in which hemp affects the body. (It’s important to note that hemp cultivation is still banned in Idaho and South Dakota.) To be considered “hemp,” a cannabis plant can contain no more than 0.3% THC.

When it comes to medical marijuana, CBD takes center stage as the plant’s most helpful cannabinoid. Derived directly from hemp, CBD interacts with your body’s endocannabinoid system (ECS). According to a 2017 study published on ScienceDirect, the ECS is “involved in many physiological regulation pathways in the human body,” such as mood, memory, appetite, and pain sensations.

ECS receptors and neurotransmitters are lipid-based, which is why CBD extract is often housed in some type of oil for greater effectiveness. The two primary ECS receptors are CB1 and CB2, and THC easily binds to both receptors. CBD, on the other hand, interacts with the ECS in a slightly different manner: The cannabinoid acts as a sort of influencer, modifying a receptor’s ability to bind to other cannabinoids.

Yet cannabinoids don’t work alone when it comes to effects on the body. The hemp plant contains a number of other components that may help determine treatment efficacy, such as terpenes. Like cannabinoids, terpenes are developed in the trichomes of the hemp plant, and are primarily responsible for producing the unique scent of a particular strain. Terpenes also play a role in a plant’s medicinal effects. For example, the limonene terpene (also found in lemons) may improve mood and reduce anxiety. 

Efficacy and Medical Uses for CBD

Reducing anxiety, in fact, is one of the most lauded effects of CBD. Studies indicate that CBD may directly activate the 5-HT1A serotonin receptor, inducing anti-anxiety and antidepressant effects. Therefore, CBD could ultimately replace a number of pharmaceutical medications, many of which come with negative side effects and may be addictive.

For its part, CBD is considered non-addictive and may also help with pain relief. To wit, researchers have proposed the use of CBD as an alternative to opioids for managing chronic pain. Opioid misuse has become a scourge on society in recent years, and preliminary data indicates that “opioid overdoses and abuse have been significantly lower in states that have legalized cannabinoids,” writes the Psychiatric Times. However, that data is in dispute, and more research is needed.

For now, we can at least focus on the proven benefits of CBD, including the reduction of seizure frequency on some patients. According to the American Epilepsy Society, CBD has been shown to reduce seizures among patients with treatment-resistant epilepsy, but CBD treatment effectiveness may wane over time, requiring higher doses. CBD works against seizures by conferring neuroprotective effects to the hippocampus, and side effects of the treatment may include nausea and vomiting. 

How CBD is Becoming a Mainstream Treatment Option

But the benefits of CBD may not be accessible to everyone. Despite the ubiquity of CBD and hemp shops across the U.S., many patients still lack access to the versatile cannabinoid. Although hemp is federally legal, many state legislators still have difficulty differentiating between its components, continuing to lump THC and CBD into the same category of dangerous substances, nearly 100 years after the implementation of the Marihuana Tax Act.

On a public health scale, the legal status of CBD is problematic. Ensuring high-quality care across diverse populations hinges on the concept of ranges — ranges of populations, conditions, choices, where both providers and patients can work together with a variety of options to create personalized treatment plans. Especially for those patients who have tried other methods without success, access to CBD may be paramount to long-term healing.

We still have a long way to go before the entire nation has access to alternative treatments like CBD and its potential benefits. Despite legal roadblocks, plenty of research on cannabidiol is being conducted, including the creation of enzymatic networks in yeast strains. As such, CBD promises to be an exciting component of the medical landscape well into the future.

(http://www.pharmamicroresources.com/)

Wednesday, 25 March 2020

Inner 'clockwork' sets the time for cell division in bacteria


The ability of pathogens to multiply in the host is crucial for the spread of infections. The speed of bacterial division greatly depends on the environmental conditions. Under unfavorable conditions, such as nutrient deficiency, bacteria tend to pause after division and reproduce more slowly. But how do bacteria know, when it is time to enter the next round of cell division?

A team at the Biozentrum of the University of Basel, led by Prof. Urs Jenal has now identified a central switch for reproduction in the model bacterium Caulobacter crescentus: the signaling molecule c-di-GMP.


Under favorable living conditions, newborn bacteria begin to produce the signaling molecule -- this starts the clock ticking. The initially low c-di-GMP level activates a first kinase. This activates the expression of over 100 genes, which drive the cell towards division and boost the production of c-di-GMP.


The resulting peak levels of c-di-GMP finally stimulate the last wheel of the machinery, also a kinase.

See:

Andreas Kaczmarczyk, Antje M. Hempel, Christoph von Arx, Raphael Böhm, Badri N. Dubey, Jutta Nesper, Tilman Schirmer, Sebastian Hiller, Urs Jenal. Precise timing of transcription by c-di-GMP coordinates cell cycle and morphogenesis in Caulobacter. Nature Communications, 2020; 11 (1) DOI: 10.1038/s41467-020-14585-6

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

Tuesday, 24 March 2020

Interesting flu facts


Imagine this:

It’s an uncomfortably cold day outside, but an emergency forces you to go outside underdressed for the temperature. And… your hair is still wet.

Are you more likely to catch the flu this way?

Alarms.org's new flu guide answers this and other widely-held “myths”: https://www.alarms.org/flu-statistics/

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

Monday, 23 March 2020

Analytical method transfer and the supplier change processes for Bacterial Endotoxin Testing



LAL testing is an established part of many QC microbiology laboratories, and subject to the similar test protocols and assay requirements as with other analytical methods, albeit with the wider acceptance criterion that reflects the test’s status as a biological assay. With such laboratory methods there will be times when a laboratory will either need to transfer an existing method between one laboratory an another or where the core reagent needs to be changed; both scenarios require a reassessment of previously assessed samples. This article, in considering analytical method changes, uses the LAL test (specifically a photometric test method) as an example of how a method can be transferred.

In relation to this, Tim Sandle and Kerry Skinner have written an article. Here is the abstract:

A key objective is with any method transfer or change is ensuring that product and process knowledge is captured. Challenges with this process can arise because of poor analytical method robustness; where differences in analytical technology that have not been duly considered; due to different working cultures; or where there are differences in ways of working between laboratories or alterations that are required in relation to different methodologies. Such issues are more likely when a method is transferred but problems can also arise when a reagent changes, especially where the replacement reagent is inferior. The important aspect will both scenarios is in taking time to plan the change process.


The reference is:

Sandle, T. and Skinner, K. (2019) Analytical method transfer and the supplier change processes for Bacterial Endotoxin Testing, European Pharmaceutical Review, 24 (5): 54-57

The article can be accessed here: https://www.europeanpharmaceuticalreview.com/article/103589/european-pharmaceutical-review-issue-5-2019/

For further details, contact Tim Sandle

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

Sunday, 22 March 2020

Antioxidant in green tea could help fight TB


An antioxidant found in green tea plant could help in fighting tuberculosis. The compound is epigallocatechin gallate and it can inhibit the growth of a tuberculosis-causing bacteria strain. This may pave the way for the development of novel drugs.

The development comes from Nanyang Technological University, Singapore (Singapore), where scientists have demonstrated that the antioxidant called epigallocatechin gallate can inhibit the growth of the Mycobacterium tuberculosis. The researchers have shown that the compound binds to an enzyme (ATP synthase) that provides energy to bacterial cells. When pigallocatechin gallate becomes attached to the enzyme, the available energy for critical processes required for bacterial cell division is reduced, leading to inhibition.

Tuberculosis is one of the most widespread bacterial diseases on the planet. It is an infection that has plagued humans for over millennia. Symptoms of infection from this bacterial disease include a chronic cough with blood-tinged sputum, fever, night sweats, and weight loss.

Although global health efforts are proving effective and the incidence of the tuberculosis are falling, based on World Health Organization data, drug-resistant tuberculosis is becoming a global problem.

In terms of the significance of the research, if it can be determined areas on the enzyme where the compound binds and dampens energy production, then it could be possible to develop a drug product to help fight the bacteria and prevent the course of an infection in a patient.

Speaking with Laboratory Roots, lead researcher Professor Gerhard Grüber says: "Though tuberculosis is curable, the success of current drugs on the market is increasingly being overshadowed by the bacteria's clinical resistance. Our discovery of the EGCG's ability to inhibit the growth of M. tuberculosis will allow us to look at how we can improve the potency of this compound in green tea, and other similar compounds, to develop new drugs to tackle this airborne disease."

The research has been published in the journal Scientific Reports. The research paper is titled "Disrupting coupling within mycobacterial F-ATP synthases subunit ε causes dysregulated energy production and cell wall biosynthesis."

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

Saturday, 21 March 2020

New Bacteriophage-based Therapy for the Treatment of Bacterial Vaginosis


Bacterial vaginosis (BV) is the most common vaginal disorder worldwide in women of the reproductive age and is associated with the increased risk of upper reproductive tract infections, adverse pregnancy outcomes, and high susceptibility to sexually transmitted infections.

Currently antibiotics are the mainstay of therapy for BV, however, the recurrence rate is up to 70 % within a year. BV is characterized by a microbial imbalance of the vaginal flora, predominantly the loss of normally dominant Lacobacilli and the overgrowth of anaerobic bacteria. More than a dozen bacterial species have been identified to be associated with BV, among them species of the genus Gardnerella were found to be the most prevalent, and without them BV symptoms seem not to occur.

PhagoMed isolated a panel of bacteriophage endolysins, highly evolved hydrolytic enzymes, that specifically degrade the bacterial cell wall of all tested species within the genus Gardnerella. By genetic engineering the lytic activity of these endolysins was further enhanced whereat the specificity for Gardnerella spp. was still preserved.

When testing these endolysins on different Lactobacilli strains their viability was not affected.
This combination of high selectivity as well as high effectiveness in killing Gardnerella makes endolysins a very attractive alternative to antibiotics for the treatment of BV.

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

Friday, 20 March 2020

Harnessing RNA sensors inhibits growth of tuberculosis bacterium


New research indicates enhancing human body cells so that they are more attuned to killing invasive Mycobacterium tuberculosis is an effective means for controlling the spread of the disease. The approach involves harnessing RNA sensors, which detect the RNA of invading pathogens. The research is also of wider pathology interest since RNA sensor molecules were previously thought to be involved in fighting viruses and not bacteria; the new study shows their role in combating invasive bacteria.

Tim Sandle has written an article for Infectious Disease Hub.

Here is an extract:

The new research strand aims to trigger human cells to become effective at killing M. tuberculosis cells by harnessing RNA sensors. The aim is to enhance the detection of invading pathogens and to increase the possibility of the body’s immune system in killing the invaders before they can secrete effector proteins. RNA sensors are part of the innate immune system and they are initiated by recognition of pathogen-associated molecular patterns. Such recognition initiates signalling cascades that culminate in a coordinated intracellular innate immune response designed to control infection.

The reference is:

Sandle, T. (2019) Harnessing RNA sensors inhibits growth of tuberculosis bacterium, Infectious Disease Hub, November 2019. At: https://www.id-hub.com/2019/11/26/harnessing-rna-sensors-inhibits-growth-tuberculosis-bacterium/

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

Thursday, 19 March 2020

New research outlines advice for coping with acne


Acne is a common skin condition, affecting people at different ages, and one that can wreak havoc with your self-esteem and often appears at the worst possible time. New research identifies some strategies for dealing with the condition.

To help to increase the knowledge around the condition of acne, the website eMediHealth has completed an in-depth survey about the skin condition acne. To arrive at the findings, 64 dermatologists participated in the research (including those based in the U.S., U.K. Canada and Australia). The research output reveals some interesting facts about acne, as well as common mistakes people make while attempting to deal with the condition.

Acne vulgaris refers to a long-term skin disease, which arises when hair follicles become clogged with dead skin cells and oil from the skin. There is also a probable association with the anaerobic bacterium Cutibacterium acnes, in that increased sebum production creates an environment that can sustain the colonization of the bacterium. The condition is often a source of discomfort for people and it is connected with mental health issues, such as low self-esteem.

Why acne occurs

The research produced a number of interesting findings about acne. The first relates to the most common skin type associated with the condition. Here the overwhelming majority (92.1 percent) of the dermatologists connected the condition to oily skin. This is because those with oily skin tend to have overactive oil glands that produce excessive sebum.

As to why certain people have a tendency to develop acne, hormonal imbalance was stated to play a major role in the development of acne and was identified as the top trigger for acne, Other causative factors were identified as high levels of stress and excessive sugar intake.

However, other factors that have been commonly associated with the development of acne, like smoking (20.31 percent), pollution (17.18 percent), and dust (7.81 percent), were rated low by the experts.

In terms of the after-effects of acne, more than half (55 percent) of the participants in the study held the view that the risk of scarring largely depends on the type of acne, and 28 percent believed that it depends on both the type of acne and the medication used to treat it. Generally, inflammatory acne is viewed as more likely to lead to scarring compared with non-inflammatory acne.

Dealing with acne

In terms of strategies for dealing with acne, ensuring that all make-up is thoroughly removed was found to be important. With this issue, 70.31 percent of the dermatologists emphasized the importance of removing every trace of makeup before a person with acne goes to bed. This is necessary so that the skin can breathe easily while the person sleeps.

Another factor to take into consideration for minimizing acne relates to skin cleaning. 78.13 percent cited that excessive skin cleaning, along with scrubbing, represents a common mistake made by patients with acne.

Where acne is severe, the dermatologists stressed the importance of seeking advice. With this issue, 79.69 percent said that too many patients with acne usually make the error of not seeking dermatological help, which can worsen their condition.

With standard acne treatments, isotretinoin and retinoids (medications that are derived from vitamin A) emerged as clear favorites among the dermatologists, each registering 75 percent of the votes.

As a word of warning for those with acne considering self-medicating, the overall effectiveness of home remedies for acne was deemed to be low. This means those looking for quick solutions through unverified treatments or remedies should take care. A total of 46.88 percent of the dermatologists stated that none of the ‘alternative’ remedies are likely to yield any significant results, and only 3.13 percent felt that the listed acne remedies may be helpful in some way.

Of these alternatives, witch hazel topped the list with 17.9 percent of the votes, followed by essential oils, which were recommended by 15.63 percent of the respondents.

For people with mild acne, the advice is to speak to a pharmacist or seek advice from a dermatologist about the appropriate medicines to treat it. For those with severe acne, advice from a medical practitioner should be sought.

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

Wednesday, 18 March 2020

FDA: Chemistry, Manufacturing, and Control (CMC) Information – gene therapy


The FDA has issued a new guidance document titled “Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)”.

Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use.


FDA generally considers human gene therapy products to include all products that mediate their effects by transcription or translation of transferred genetic material or by specifically altering host (human) genetic sequences.  Some examples of gene therapy products include nucleic acids (e.g., plasmids, in vitro transcribed ribonucleic acid (RNA)), genetically modified microorganisms (e.g., viruses, bacteria, fungi), engineered site-specific nucleases used for human genome editing, and ex vivo genetically modified human cells.

For details see: https://www.fda.gov/media/113760/download

Posted by Dr. Tim Sandle, Pharmaceutical Microbiology Resources (http://www.pharmamicroresources.com/)

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